1.1 Pemigatinib is recommended, within its marketing authorisation, as an option for treating locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after systemic therapy in adults. It is recommended only if the company provides pemigatinib according to the commercial arrangement.
Why the committee made these recommendations
Current treatment for advanced cholangiocarcinoma with an FGFR2 fusion or rearrangement that has progressed after systemic therapy is symptom control, with or without modified folinic acid, 5‑fluorouracil and oxaliplatin (mFOLFOX) chemotherapy.
Clinical evidence from 1 study suggests that pemigatinib may be more effective than current treatments. This is uncertain because the study did not directly compare pemigatinib with symptom control or mFOLFOX. But the cancer is rare. This means the number of people who could take part in a study is small, making it difficult to collect robust comparative data. So, the uncertainty is considered acceptable.
Pemigatinib meets NICE's criteria for a life-extending treatment at the end of life. The cost-effectiveness estimates are uncertain but are likely to be within the range that NICE considers a cost-effective use of NHS resources. So, pemigatinib is recommended.